Novartis Confirms Plans to File BLA for CAR T-Cell Therapy During Early 2017

Novartis said it is on track to file a biologics licence application (BLA) with FDA during early 2017 for use of its investigational chimeric antigen receptor T-cell (CAR T) therapy, CTL019, in relapsed/refractory (r/r) pediatric patients and young adults with B-cell acute lymphoblastic leukemia. Submission to the European Medicines Agency (EMA) for approval of the drug in Europe is expected later in 2017.

Confirmation of the proposed CTL019 filing timelines follows the release of positive interim data from the global, registrational Phase II ELIANA study, which showed that 41 out of 50 (82%) patients achieved complete remission, or complete remission with incomplete blood count recovery, at three months post infusion with CTL019. No residual disease was observed in any patient with complete remission. Estimated relapse-free rate among responders was 60%, six months after infusion. Forty eight percent of enrolled patients experienced grade 3 or 4 cytokine release syndrome (CRS), but there were no CRS-related deaths.

Novartis says ELIANA is the first pediatric global CAR T-cell registrational trial to have enrolled across 25 centers in the US, EU, Canada, Australia and Japan. The study is supported by the US-based CTL019 study ENSIGN, and earlier sigle site trials in r/r pediatric and young adult patients with B-cell acute lymphoblastic leukemia.

"This first-of-its-kind trial represents exciting progress toward our goal of helping children and young adults with relapsed or refractory B-cell ALL, a patient population with an urgent need for new treatment options," stated Bruno Strigini, CEO, Novartis Oncology. "We are committed to advancing CTL019 and look forward to working closely with the FDA and EMA in the coming months." The ELIANA trial data were to be presented today at the 58th American Society of Hematology (ASH) annual meeting.

The Novartis trial data come just 24 hours after Kite Pharma reported positive data from a preliminary analysis of its Phase I ZUMA-3 and ZUMA-4 trials investigating the CAR T-cell therapy candidate, KTE-C19, in 13 adult and pediatric patients with r/r acute lymphoblastic anemia. The results showed that 82% of evaluable patients (9 of 11) achieved complete remission or complete remission with incomplete or partial haematological recovery, with all 11 evaluable responders testing negative for minimal residual disease. Five of 13 (38 percent) of patients had ≥ grade 3 CRS, and there was also one death from KTE-C19-related CRS. Kite said it plans to start the Phase II stages of the trials during 2017.